A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Study Summary

This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose.

Conditions

Glycogen Storage Disease Type Ia

Interventions / Drugs

BEAM-301: Single dose of BEAM-301 administered by IV

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment36 participants

Primary Completion—

Lead SponsorBeam Therapeutics Inc.

Data Retrieved2026-04-11 04:07:17.424756+00:00

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